NEJM: Emapalumab Clinical Trial Shows Positive Results Against HLH

Research By Michael Jordan, MD

Post Date: May 7, 2020 | Publish Date: May 6, 2020


A laboratory technician at Cincinnati Children’s conducts immunology testing for the severe childhood immune disorder HLH (hemophagocytic lymphohistiocytosis), which damages vital organs and tissues. Researchers report in the New England Journal of Medicine treating patients with a new drug that saved kid’s lives with less toxicity and fewer side effects. Doctors hope the treatment will help dent HLH’s high mortality rate.

“For the first time we have a truly targeted way to treat HLH and a drug with very low toxicity.”

—Michael Jordan, MD

Clinical trial results published in the New England Journal of Medicine show that using emapalumab to treat patients with hemophagocytic lymphohistiocytosis (HLH) saved lives with less toxicity and fewer side effects.

The results represent a significant achievement in a 20-year journey to battle HLH. This study suggests that emapalumab—the first drug ever approved for HLH by the U.S. Food and Drug Administration—when  administered with dexamethasone should become the standard of care for ‘second line’ treatment of HLH, Jordan says.

Read the Cincinnati Children’s announcement

Publication Information

Original Title:Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis
Published in:New England Journal of Medicine
Publish date:May 6, 2020

Read the Study

Research By

  • Michael Jordan, MD

    Michael Jordan, MD

    Division of Bone Marrow Transplantation and Immune Deficiency

    Dr. Jordan's research includes seeks a better understanding of histiocytic disorders and to develop novel therapies for them.